Clinical trials: Muscle Disorders
Rheumatoid Arthritis, Systemic Lupus Erythematosus
Study of Families With Twins or Siblings Discordant for Rheumatic Disorders
This study will examine families in which one sibling of a sibling pair, or twin pair, has developed a systemic rheumatic disease and one has not, to see if and how the two differ in the following: Blood cell metabolism; Types of…
- Conditions
- Rheumatoid Arthritis, Systemic Lupus Erythematosus, Idiopathic Inflammatory Myopathies
- Trial name
- NCT00055055
- Where
- 3 sites in United States
- Volunteers
- 1,056 people
- Sponsor
- National Institute of Environmental Health Sciences (NIEHS)
Duchenne Muscular Dystrophy
Observational Study of Patients With Duchenne Muscular Dystrophy Theoretically Treatable With Exon 53 Skipping
PreU7-53 is a natural history study. The objective is to monitor the clinical and radiological course of upper limb muscle impairment in patients with Duchenne Muscular Dystrophy (DMD), potentially treatable with AAV-mediated…
- Trial name
- pre U7-53 (NCT01385917)
- Where
- 2 sites in France, United Kingdom
- Volunteers
- 45 people
- Sponsor
- Genethon
Dermatopolymyositis, Interstitial Lung Disease
Pirfenidone in Progressive Interstitial Lung Disease Associated With Clinically Amyopathic Dermatomyositis
Interstitial lung disease (ILD) is a common complication of dermatomyositis (DM) with prevalence up to 65%, and is considered to be one of the determining factors of prognosis.
- Trial name
- NCT02821689
- Testing
- Pirfenidone
- Where
- China
- Volunteers
- 57 people
- Sponsor
- RenJi Hospital
Performance, Energy
Kinesiology Taping in Duchenne Muscular Dystrophy: Effects on Performance, Gait Characteristics, and Energy Consumption
Investigators investigated that the effects of kinesilogy taping on performance, energy consumption and gait characteristics in patients with Duchenne Muscular Dystrophy
- Conditions
- Performance, Energy, Gait, Duchenne Muscular Dystrophy
- Trial name
- NCT03529240
- Testing
- Kinesiology Taping
- Where
- Turkey (Türkiye)
- Volunteers
- 45 people
- Sponsor
- Hacettepe University
Hypermetabolism Due to Defect in Mitochondria, Luft Disease
A Single Case Study of Hypermetabolism
In the Metabolic Disorder clinic at The Hospital for Sick Children, the investigators identified a male, proband with possible Luft's disease.
- Trial name
- NCT04805268
- Testing
- 18F FDG PET/CT
- Where
- Canada
- Volunteers
- 1 people
- Sponsor
- The Hospital for Sick Children
Neuroblastoma, Rhabdomyosarcoma
Alpha/Beta T and B Cell Depletion With Zoledronic Acid for Solid Tumors
Hematopoietic stem cell transplantation can cure patients with blood cancer and other underlying diseases. αβ-T cell and B cell depletion has been introduced to decrease GVHD and PTLD and has demonstrated effectiveness for…
- Conditions
- Neuroblastoma, Rhabdomyosarcoma, Synovial Sarcoma, Peripheral Nerve Sheath Tumors
- Trial name
- NCT06625190
- Testing
- Miltenyi CliniMACS Prodigy ® system, Zoledronic acid
- Where
- United States
- Volunteers
- 27 people
- Sponsor
- University of Florida
Cystic Fibrosis
Fitness, Body Composition, Physical Activity and Sleep Patterns in People With Cystic Fibrosis on Modulator Therapy.
Cystic fibrosis (CF) is the most common inherited condition in the United Kingdom, affecting approximately 10,837 people. It is well recognised that regular exercise is clinically important for people with CF.
- Trial name
- Understand-CF (NCT05857709)
- Where
- United Kingdom
- Volunteers
- 100 people, healthy volunteers welcome
- Sponsor
- University of Portsmouth
Chronic Pain Syndrome, Fibromyalgia
Assessing the Impact of Exercise Based Intensive Interdisciplinary Pain Treatment (IIPT) on Endogenous Pain Modulation in Youth With Chronic Pain Syndromes
This work will answer two critical questions: 1) Does intensive interdisciplinary pain treatment (IIPT) involving aerobic exercise help normalize pain processing in youth with chronic pain syndromes and 2) Are aerobic fitness…
- Conditions
- Chronic Pain Syndrome, Fibromyalgia, Chronic Headache Disorder, Functional Abdominal Pain Syndrome
- Trial name
- NCT05491499
- Where
- United States
- Volunteers
- 43 people
- Sponsor
- Boston Children's Hospital
Limb Girdle Muscular Dystrophy Type 2C, Gamma-sarcoglycanopathy
Clinical Study of AAV1-gamma-sarcoglycan Gene Therapy for Limb Girdle Muscular Dystrophy Type 2C
The purpose of this trial is to study the evaluation of clinical safety and feasibility of gene therapy in patients with limb girdle muscular dystrophy type 2C (gamma-sarcoglycanopathy).
- Trial name
- NCT01344798
- Testing
- AAV1-gamma-sarcoglycan vector injection
- Where
- France
- Volunteers
- 9 people
- Sponsor
- Genethon
Relapsed Pediatric Solid Tumors, Refractory Pediatric Solid Tumors
MR-guided High Intensity Focused Ultrasound (HIFU) on Pediatric Solid Tumors
The purpose of this study is to determine if Magnetic Resonance guided High Intensity Focused Ultrasound ablative therapy is safe and feasible for children, adolescents, and young adults with refractory or relapsed solid tumors.
- Conditions
- Relapsed Pediatric Solid Tumors, Refractory Pediatric Solid Tumors, Rhabdomyosarcoma, Ewing Sarcoma
- Trial name
- NCT02076906
- Testing
- Magnetic Resonance High Intensity Focused Ultrasound
- Where
- 2 sites in United States
- Volunteers
- 7 people
- Sponsor
- AeRang Kim
Duchenne Muscular Dystrophy
A Registered Cohort Study on Duchenne Muscular Dystrophy
Dystrophinopathy is a term of X-linked recessive genetic disease, including Duchenne Muscular Dystrophy, Becker Muscular Dystrophy, and the X-linked dilated cardiomyopathy.
- Trial name
- NCT04012671
- Where
- China
- Volunteers
- 2,000 people
- Sponsor
- Ning Wang, MD., PhD.
Duchenne Muscular Dystrophy
Safety and Tolerability of WVE-210201 in Patients With Duchenne Muscular Dystrophy
This is a Phase 1, double-blind, placebo-controlled, single ascending dose cohort study to evaluate the safety, tolerability, and plasma concentrations of WVE-210201 in ambulatory and non-ambulatory male pediatric patients with…
- Trial name
- NCT03508947
- Testing
- WVE-210201
- Where
- 13 sites in United States, Belgium and 5 more
- Volunteers
- 36 people
- Sponsor
- Wave Life Sciences USA, Inc.
X-linked Myotubular Myopathy
A Long-term Follow-up Study in Male Participants With X-linked Myotubular Myopathy Treated With ASP2957
X-linked myotubular myopathy (XLMTM) is a rare and serious condition present at birth where the muscles do not work properly. There are currently no approved therapies for XLMTM.
- Trial name
- NCT07876804
- Testing
- ASP2957
- Volunteers
- 9 people
- Sponsor
- Astellas Gene Therapies
Scapular Dyskinesis, Scapuloshoulder Disorders
Relation Between Swimming Stroke Types And Scapular Dyskinesia In Swimmers: A Cross - Sectional Study
The goal of this cross-sectional observational study is to examine the relationship between swimming stroke specialization and scapular movement and shoulder function in healthy competitive swimmers aged 13 to 19 years.
- Trial name
- NCT07859826
- Where
- Egypt
- Volunteers
- 80 people, healthy volunteers welcome
- Sponsor
- Nouran Emad Hussin Elbosaily
Fibromyalgia
Coping Skills Training for Adolescents With Fibromyalgia
Juvenile fibromyalgia is a chronic pain condition that can cause considerable suffering and difficulty in an adolescent's day-to-day activities.
- Trial name
- NCT00086047
- Testing
- Coping Skills Training, Education
- Where
- 3 sites in United States
- Volunteers
- 114 people
- Sponsor
- Children's Hospital Medical Center, Cincinnati
Mitochondrial Diseases
EPI-743 for Mitochondrial Respiratory Chain Diseases
This study will evaluate the safety and efficacy of EPI-743 in participants with severe mitochondrial respiratory chain diseases who are considered to be within 90 days of end-of-life care.
- Trial name
- NCT01370447
- Testing
- EPI-743
- Where
- 16 sites in United States
- Sponsor
- PTC Therapeutics
Duchenne Muscular Dystrophy
Duchenne Muscular Dystrophy Tissue Bank for Exon Skipping
We will utilize the Cooperative International Neuromuscular Research Group (CINRG) network to collect and store tissue and blood from patients with Duchenne muscular dystrophy (DMD) with specific genetic mutations within the…
- Trial name
- NCT01772043
- Where
- 9 sites in United States, Canada
- Volunteers
- 53 people
- Sponsor
- Cooperative International Neuromuscular Research Group
Primary Body Region (Arranged Most Common to Least), Lumbar/SI
ATI Evidence-based Guide Investigating Clinical Services
The goal of this study is to evaluate how standard-of-care outpatient rehabilitation is delivered and how variation in care delivery mechanisms relates to clinical outcomes, service utilization, and value in patients receiving…
- Conditions
- Primary Body Region (Arranged Most Common to Least), Lumbar/SI, Knee, Shoulder
- Trial name
- AEGIS (NCT02285868)
- Where
- United States
- Volunteers
- 4,000,000 people, healthy volunteers welcome
- Sponsor
- ATI Holdings, LLC
Muscular Atrophy, Spinal
Investigate Safety, Tolerability, PK, PD and Efficacy of Risdiplam (RO7034067) in Infants With Type1 Spinal Muscular Atrophy
Open-label, multi-center clinical study is to assess the safety, tolerability, pharmacokinetic (PK), pharmacodynamics (PD), and efficacy of Risdiplam (RO7034067) in infants with Type 1 spinal muscular atrophy (SMA).
- Trial name
- FIREFISH (NCT02913482)
- Testing
- Risdiplam
- Where
- 32 sites in United States, Belgium and 14 more
- Volunteers
- 62 people
- Sponsor
- Hoffmann-La Roche
Spinal Muscular Atrophy
A Review of the Management and Outcomes of Children With SMA in the West Midlands During 2017-2022
Spinal Muscular Atrophy (SMA) is a rare neuromuscular condition, characterised by loss of motor neurons as a result of a mutation in the survival motor neuron gene.
- Trial name
- NCT05994950
- Where
- 2 sites in United Kingdom
- Volunteers
- 30 people
- Sponsor
- Robert Jones and Agnes Hunt Orthopaedic and District NHS Trust
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